Stem Cell Research Focusing on Autism’s Genetic Mysteries Earns $2.125 Million Grant at Robert Wood Johnson Medical School

PISCATAWAY — The social symbol for autism awareness, a ribbon of brightly-colored puzzle pieces, reflects the complexity of Autism Spectrum Disorder (ASD). A new five-year $2.125 million grant from the New Jersey Governor’s Council for Medical Research and Treatment of Autism will fund research of induced pluripotent stem cells that may be used to piece together the genetic pathways of autism and lead to new treatments for individuals affected by ASD.

The research, led by James H. Millonig, PhD, associate professor of neuroscience and cell biology at the University of Medicine and Dentistry of New Jersey-Robert Wood Johnson Medical School, is being conducted as part of the Robert Wood Johnson Medical School New Jersey Autism Center of Excellence (NJ ACE).

“Autism is defined by a spectrum of behavioral and neurological abnormalities, with distinct characteristics for each individual denoting that there are multiple underlying genetic causes,” said Dr. Millonig, who also is assistant dean of medical science training at Robert Wood Johnson Medical School and a member of the Center for Advanced Biotechnology and Medicine, a joint institute of the medical school and Rutgers, The State University of New Jersey. “Working with my colleagues in neuroscience and at the Child Health Institute of New Jersey and Rutgers, we hope to identify the neurobiological, molecular and genetic basis – the biological signature – of autism.”

Autism is a disorder of the brain, which is mostly comprised of neurons. Induced pluripotent stem cells (iPSCs) are scientifically-derived stem cells from individuals that mimic the traits of embryonic stem cells and can be used to create other cell types. Therefore, the research team will use iPSCs to create human neurons in order to understand how they develop abnormally in individuals with autism. The scientists will then test FDA-approved drugs on the neurons to identify therapeutic treatments that may improve or reverse the disorder.

“We know of a lot of genes associated with autism, but don’t know when, where and how they act in development,” explains Dr. Millonig. “We need to look at neurons and determine how they mature differently in order to develop better drug therapies that are tailored to the needs of individuals with autism.”

The multi-institutional NJ ACE team includes Emanuel DiCicco-Bloom, MD, professor of neuroscience and cell biology and pediatrics at the medical school; Linda M. Brzustowicz, MD, professor of genetics, Rutgers University; Chi-wei Lu, PhD, assistant professor of obstetrics, gynecology, and reproductive sciences and Zhiping Pang, PhD, assistant professor of neuroscience and cell biology — both at the Child Health Institute of New Jersey; and Yong Lin, PhD, at The Cancer Institute of New Jersey. The Child Health Institute of New Jersey and The Cancer Institute of New Jersey are Centers of Excellence at Robert Wood Johnson Medical School. Consultants on the grant include the Rutgers University Cell and DNA Repository; Ronald Hart, PhD, professor of cell biology and neuroscience, Rutgers University; Bonnie Firestein, PhD, professor of cell biology and neuroscience, Rutgers University; Jennifer Moore, PhD, associate director, National Institute of Mental Health Stem Cell Center, and research assistant director, Stem Cell Research Center, Rutgers University.